ДЕНАРО Мария Джоанн,ФАЙНЕР Митчел Ховард,ВЕРЕС Габор
申请号：
RU2014100160
公开号：
RU2014100160A
申请日:
2012.06.08
申请国别(地区):
RU
年份:
2015
代理人:
摘要：
1. A vector including: (a) left (5 ′) retroviral LTR； (b) central polypurine DNA / flap region of DNA (cPPT / FLAP)； (c) retroviral export-regulating element； (e) promoter active in the microglia cell functionally linked to a polynucleotide encoding a polypeptide of the ATP-binding cassette transporter of the subfamily D (ALD), member 1 (ABCD1), and (f) the right (3 ′) retroviral LTR； this vector does not include the post-transcriptional regulatory element of the marmot hepatitis virus (WPRE ) .2. The vector according to claim 1, where the specified vector is a lentiviral vector. The vector according to claim 2, wherein said lentivirus is HIV. The vector according to claim 2, where the specified lentivirus is HIV-1.5. The vector according to claim 1, wherein the 5′-LTR promoter is replaced by a heterologous promoter. The vector according to claim 5, wherein said heterologous promoter is a cytomegalovirus (CMV) promoter, a Routh sarcoma virus (RSV) promoter, or a promoter of a vacuumed monkey virus (SV40). The vector of claim 5, wherein said heterologous promoter is a CMV.8 promoter. The vector of claim 1, wherein said 5′-LTR or 3′-LTR is a lentiviral LTR. The vector of claim 1, wherein said 5′-LTR and 3′-LTR are lentiviral LTR. The vector of claim 8 or 9, wherein said lentivirus is HIV-1.11. The vector of claim 10, wherein said 3′-LTR includes one or more modifications. The vector of claim 10, wherein said 3′-LTR includes one or more deletions. The vector of claim 10, wherein said 3′-LTR is an LTR of a self-inactivating vector. The vector according to claim 1, wherein said retroviral export-regulating element is a Rev-sensitive regulatory element (RRE). All1. Вектор, включающий:(a) левый (5′) ретровирусный LTR；(b) центральный полипуриновый участок/флэп ДНК (cPPT/FLAP)；(c) ретровирусный, регулирующий экспортирование элемент；(e) промотор, активный в клетке микроглии, функционально связанный с полинуклеотидом, кодирующим полипептид АТФ-связывающего кассетного транспорт
